Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: Progress and challenges

Current Gene Therapy

Volumn 12, Issue 3, 2012, Pages 152-160

  Arechavala Gomeza, Virginia ^a   Anthony, Karen ^a   Morgan, Jennifer ^a   Muntoni, Francesco ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Antisense oligonucleotides; Becker muscular dystrophy; Clinical trials; Duchenne muscular dystrophy; Dystrophin; Exon skipping; RNA therapy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; BETA DYSTROGLYCAN; DRISAPERSEN; DYSTROPHIN; ETEPLIRSEN; MYOD PROTEIN; PLACEBO;

BECKER MUSCULAR DYSTROPHY; DISEASE SEVERITY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE MUTATION; HUMAN; IMMUNE RESPONSE; MUSCLE BIOPSY; MUSCLE CRAMP; MUSCLE WEAKNESS; MYALGIA; NONHUMAN; OPEN READING FRAME; REVIEW; RNA SPLICING; TREATMENT RESPONSE;

ALTERNATIVE SPLICING; ANIMALS; DYSTROPHIN; EXONS; GENETIC THERAPY; HUMANS; MICE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; OLIGONUCLEOTIDES, ANTISENSE; RNA PRECURSORS;

EID: 84862625633     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/156652312800840621     Document Type: Review

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